Session 1


Outcomes of the 2008 Conference and work of the Access to Medicines Working Group

Mr David Learmonth, Deputy Secretary, Department of Health and Ageing
Mr Will Delaat, Chairman, Medicines Australia

Keynote Address

Health Technology Assessment for future generations

SPEAKER Professor Lloyd Sansom AO, Emeritus Professor, Division of Health Sciences, University of South Australia

Session 2

Future challenges to Health Technology Assessment

TUESDAY, 30 AUGUST • 11:00 AM–1:00 PM

CHAIR Dr Rohan Hammett, National Manager, Therapeutic Goods Administration

Australia has a mature HTA system. However, many medicines that are currently in R&D pipelines are potentially biologically targeted therapies. This session will examine the challenges posed both locally and internationally by the more complex trial designs in the context of medicines in development globally. Discussion of these issues is particularly relevant in the area of co-dependant technologies. This session will also examine the policy challenges for Australia in valuing these advancements and finally from the consumers’ perspective, the need to ensure that our health system continues to deliver ‘equity of access’.

Data requirements for the future: High cost/highly targeted therapies

Prof Kathryn A. Phillips, Professor of Health Economics and Health Services Research, University of California, San Francisco

This presentation will address the growth of biologics and co-dependent tests and treatments, the inevitable trend towards personalised medicine, and observations regarding the relevance to Australia of developments in the United States. Four key challenges and opportunities will be discussed: negotiating shifting industry paradigms, balancing innovation and regulation, building an evidence base, and determining value and reimbursement. The presentation will pay particular attention to ‘evidence’ and ‘value’, and use examples to provide insights into these issues.

Policy Challenges

Prof Robyn Ward, Clinical Associate Dean, Princeof Wales Clinical School, University of NSW

The Pharmaceutical Benefits Advisory Committee and the Medical Services Advisory Committee were established to provide advice to the Minister for Health and geing on the circumstances under which public funding for drugs, medical technologies or procedures should be supported. Over the last few years experience with the assessment of various co-dependent cancer test/drug packages has exposed many of the impediments to realising personalised medicine. This presentation will discuss examples of the health technology assessment process for a number of cancer medicines and their associated tests to illustrate the policy and practical challenges of delivering new biologically targeted therapies.

Ethics in drug development process: Societal perspectives

Ms Jennifer Doggett, Fellow, Centre for Policy Development

This presentation will draw on recent research into the needs and priorities of consumers for access to quality medicines and how these can be used to inform the developmental process for new medicines.

Session 3

Data Linkages and Consumer Engagement

TUESDAY, 30 AUGUST • 2:00–3:30 PM

CHAIR Mr Laurie Wilson, President, National Press Club

Advances in data storage and management have opened new opportunities for researchers and policy makers to link and mine data sets to deliver a range of health and economic benefits. One of the many ways to achieve this is through harnessing the potential of e- ealth; more specifically, integration of health and other data sets to provide information to improve decision making in healthcare delivery. This session will look at the experience so far with linking public data sources, examine the opportunities and barriers to achieving success and finally, from a consumer perspective, examine the importance of balancing safeguards to protect an individual’s privacy with the need for access to population health data.

Using linked data sets to improve healthcare delivery

Assoc Prof Libby Roughead, Future Fellow, School of Pharmacy and Medical Science, University of South Australia

Australia’s system of universal healthcare, funded largely by government, has resulted in the availability of significant data on healthcare utilisation that represent the whole population. These data can be used in a number of ways to improve healthcare from consistency of care, evidence-based guidelines and evaluation of interventions to the generation of new knowledge. This presentation will include examples of the use of data for these purposes.

Sensitive health information and privacy

Mr Malcolm Crompton, Managing Director, Information Integrity Solutions P/L

Health information is sensitive. Legislation tells us that it is, and people’s feelings tell us that it is. So, how do we manage these feelings and how do we manage health data while moving forward with health research and development? Privacy requirements need not be a thorn in our side; they can be an opportunity to build communication flows with consumers.

This presentation offers thoughts about frameworks and technical solutions in Australia’s health privacy space. The presenter will draw on his global experience in privacy matters, the delivery of a large number of Privacy Impact Assessments to government and private sector entities and his experience as Privacy Commissioner of Australia.

Issues for consumers

Ms Karen Carey, Consumer Advocate

There is a constant tension between early access to new therapies and taking the time to gather sufficient evidence to know that a treatment is safe, effective and cost effective. Early access is only reasonable when it is combined with appropriate post-market surveillance, and electronic data linkage provides the best opportunity to build the level of data that will provide effective post-market surveillance. Through linking data we can find out quickly whether treatments are safe, effective and cost-effective. The wider the data collection, the more quickly trends will be disclosed. But data linkage comes with a price to consumers—privacy. The potential benefits of data linkage are great, so we need to identify, minimise and mitigate the risk of privacy impacts. In the past decade debate about these issues has helped define where the risks are greatest, who is the most vulnerable and the structure we need to ensure that the cost isn’t borne by the most vulnerable in society.

Boosting policy relevant research using linked administrative data

Prof Louisa Jorm, Foundation Professor of Population Health, University of Western Sydney

Linked administrative health data are a powerful resource for research that can drive improvements in resource allocation, service quality and health outcomes. However, relatively little use of these data is made to inform decision-making in Australia. This presentation will cover new approaches to managing data and metadata including the Secure Unified Research Environment (SURE) which has been built as part of new national infrastructure for research using linked data; methods for analysing administrative data in combination with data from other sources including cohort studies and clinical trials; and approaches to using these data to facilitate policy change, through making better use of context data and finding more persuasive ways to communicate.

Session 4

Hypothetical: A Patient’s Experience

TUESDAY, 30 AUGUST • 4:00–5:30 PM

CHAIR Prof Philip Davies, Professor of Health Systems and Policy, University of Queensland

The hypothetical ‘A patient’s experience’ provides an opportunity to gain new insights into pharmaceutical policy in Australia. By stepping into the lives of the Theticals, an imaginary Australian family, you will experience what it is like for ordinary Australians to navigate the sometimes complex world of the PBS, to struggle to figure out why we pay for prescriptions, and to try to understand why pharmaceutical companies and government sometimes just don’t see eye to eye. Your guides on this journey include leading clinicians, industry figures, academics, consumer representatives and policy specialists, all of whom will share their insights and attempt, with no preparation or foreknowledge, to tackle some of the thorny issues that patients face. It promises to be an enjoyable and informative experience.

Session 5

The Relevance of the National Medicines Policy in the Future

WEDNESDAY, 31 AUGUST • 9:00–10:30 AM

CHAIR Prof Andrew J. McLachlan, Chair, National Medicines Policy Committee; Professor of Pharmacy, University of Sydney

Australia’s National Medicines Policy has provided a useful framework, but does it meet contemporary needs? Its objectives need to be integrated and are strongly interdependent. However, tensions can and do exist between the arms of the policy. Balance is needed to harness the full potential of National Medicines Policy. This session will examine the tensions and interdependencies between the four policy objectives and explore ways of developing the Policy to achieve this balance.

Linkages between the four pillars of the NMP

Dr Lynn Weekes, Chief Executive Officer, NPS

The National Medicines Policy (NMP) aims to meet medication needs so that optimal health and economic impacts are achieved. All policy partners share responsibility to varying degrees for all four arms of the policy and for ensuring that consumers are central to all decisions. This shared responsibility is the basis for linkages and interdependencies and where the linkages are strongest so is the policy. The linkages and interdependencies of NMP partners are not without tensions and at best these are well recognised and openly debated. There are excellent examples where all partners have come together to address a medicines dilemma and been able to agree on the way forward. This ability to problem- solve has been a hallmark of the NMP partnerships working at their best.

Looking back and looking forward: How did we get here?

Prof Lloyd Sansom AO, Emeritus Professor, Division of Health Sciences, University of South Australia

It has been over 20 years since the need for a National Medicines Policy in Australia was first recognised. The seed was planted at a joint conference of the Consumers Health Forum and the Australasian Society of Clinical and Experimental Pharmacologists and Toxicologists held in Newcastle in the late 1980s. From that genesis and with support from the then Government, in particular Minister Peter Staples, came the establishment of PHARM Committee and APAC. PHARM was an expert committee and was responsible for the creation of community awareness of medicines use. APAC was a multi-representative council charged with the development of a National Medicines Policy (NMP). Throughout the 1990s both groups worked to change the culture of medicines and their use in Australia and to develop links and dialogue between stakeholders. This culminated in 2000 with the adoption of the NMP whose guiding principles are still relevant today. The delivery of healthcare and the role of medicines will continue to evolve with an ever increasing demand for goods and services and subsequent costs. The NMP must continue to form the framework for policy development and evaluation as we face these new challenges.

The importance of documentation

Dr Ross Maxwell, Procedural Rural Doctor (Queensland), and Prescribing Expert on the National Medicines Policy

Timely access to, and affordability of, world-class medicines is one of the factors contributing to the Australian population’s good health outcomes and is a key objective of National Medicines Policy. The health system has persistent cost pressures requiring continual reassessment of priorities. Consumers, health professionals, the pharmaceutical industry and government will benefit from a better understanding of the use and impact of medicines, particularly new medicines in the Australian setting. At present the health system allows robust assessment of a medicine’s efficacy and cost-effectiveness at the time of registration via the TGA and PBAC. Quality use of medicines and quality and safety of medicines is also well established as key aspects of the NMP. The recent NMP Partnership Forum examined the opportunities for and challenges of establishing a post- arket surveillance system in Australia to systematically assess medicines safety, effectiveness and cost-effectiveness across the life of a medicine.

What developments will impact on medicines policy in the future

Dr Brendan Shaw, Chief Executive, Medicines Australia

The National Medicines Policy is a balancing act between different objectives which, when aligned successfully, deliver a policy framework that ensures Australians have continued access to medicines. For industry’s part, commercial pressures, technological development, shifting growth patterns in international markets, government budgetary cutbacks and declining commercial returns are all presenting their own range of challenges and opportunities. The concern is that in the rush to economise, rationalise and prioritise the Government’s and community’s objectives, objectives of industry—so important to ensuring the medicines actually make it to market in the first place—need to be given sufficient recognition. Predictability in policy frameworks, sufficient return to justify continued investment in developing new medicines, the regulations and incentives governing research and clinical trials, opportunities for further investment in Australian operations, and the impact of reimbursement and HTA outcomes on innovation and long-term industry investment decisions are all issues that need to be considered in the context of the National Medicines Policy.

Session 6

Facilitating Access to Medicines for Special Patient Groups

WEDNESDAY, 31 AUGUST • 11:00 AM–12:30 PM

CHAIR Dr John Primrose, Medical Adviser, Pharmaceutical Evaluation Branch, Department of Health and Ageing

Using the examples of the need to deliver health outcomes to Indigenous and paediatric patients, this session will look at the challenges involved in meeting the needs of discrete populations within a PBS system that is primarily designed to meet the needs of the broader community on a population basis.

Focus on indigenous health

Prof Alan Cass, Professor, Sydney Medical School, University of Sydney and Director of the Renal and Metabolic Division, George Institute, Sydney

This presentation will explore evidence-practice gaps in the screening and management of chronic vascular disease in the Aboriginal and mainstream primary care sectors, and will discuss influences on access to necessary health care, at the health system-, service and individual-level, for Indigenous Australians with chronic diseases.

Access to medicines: Policy affecting Aboriginal peoples

Dr Sophie Couzos, Public Health Medical Officer, Aboriginal community controlled health services and the National Aboriginal Community Controlled Health Organisation

Access to essential medicines is a core element of our nation’s obligation towards the right to health for all Australians. The PBS provides capped patient copayments and a safety net scheme to improve medicines access, but disparities in access between Aboriginal and non- boriginal Australians are still substantial. A range of health programs and policies have been developed to improve access and improve quality use of medicines in remote and non-remote Australia. This presentation will describe aspects of these programs, compare and contrast, refer to success factors, and make suggestions for ways in which medicines access can be further improved.

Focus on paediatric health

Dr Sean Beggs, Paediatrician and Paediatric Clinical Pharmacologist, Royal Hobart Hospital

Children have long been disadvantaged in relation to their access to medications. The majority of medications on the Australian market have not been studied in children. It is often quoted that ‘children are not simply small adults’. Nowhere does this statement hold more true than in relation to therapeutics. The small size of the paediatric pharmaceutical market and thus lack of financial incentives means that the market often fails children. There is an obvious need and desire to address the issue in Australia. The Paediatric Medicines Advisory Group (PMAG) brings together representatives from Government, pharmaceutical industry, TGA, PBAC, paediatric pharmacists, and paediatricians to identify gaps and potential solutions while working within the current regulatory framework.

Access to medicines for special patients and target groups

Ms Deborah Waterhouse, General Manager, GlaxoSmithKline Australia and New Zealand

There are multiple challenges in meeting the needs of discrete populations within a PBS system that is primarily designed to meet broad community needs. Providing a perspective from the pharmaceutical industry, this session will address how difficulties in catering for discrete populations within the PBS act as an early indication of the challenges for delivering the medicines of the future. This highlights the need for greater flexibility within regulatory and reimbursement pathways, both to meet the needs of particular patient populations now and ensure the system can adapt in the future to make the potential of personalised medicine a reality.

Session 7

The Influence of HTA in Shaping Future Drug Development

WEDNESDAY, 31 AUGUST • 1:30–3:00 PM

CHAIR Prof Kathryn A. Phillips, Professor of Health Economics and Health Services Research, University of California, San Francisco

HTA is increasingly used around the world as a tool for assessing a medicine’s clinical and cost-effectiveness. Governments and other payers use it to make judgements about whether a medicine can be deemed ‘value for money’. Much of the discussion about medicines policy rightly focuses on ensuring that new medicines are affordable to both the consumer and taxpayer, and the policies and programs that are needed to deliver these. There is a need for serious debate, however, about how HTA based systems, in turn, are likely to shape the future drug development process; in particular, by creating incentives or disincentives to invest in specific therapeutic areas at the expense of others. This session explores how the industry may respond to market signals set by governments and payers, and how this will inform the development of medicines to meet the health needs of future generations.

Investment decisions in drug development

Dr Steve J. Romano, Senior Vice President, Pfizer, New York

While investment into pharmaceutical R&D has increased significantly over the last 10 years, the output in terms of new molecular entities reaching the market has until recently declined. When making investment decisions, most pharmaceutical manufacturers now advance new investigational compounds only when they are considered to have sufficient potential to deliver meaningful clinical differentiation to standard of care. As we investigate more complex and novel mechanisms of action in disease areas of high unmet need, dialogue must be expanded with HTA institutions and other stakeholders to align expectations regarding what evidence can be delivered in clinical development to support or accelerate access to new medicines.

Innovation dilemma: The case of antimicrobials

Prof John Turnidge, Clinical Director of Microbiology and Infectious Diseases for SA Pathology, Women’s and Children’s Hospital, Adelaide

As the world witnesses a growing number of antimicrobial-resistant bacteria causing disease, there has been a steady decline in the number of new antimicrobials being developed and reaching the market. Recently, new models for antimicrobial agent development have emerged. Ultimately though, we are caught by a paradox. New agents, especially those with novel mechanisms of action, are often kept in reserve after release, rather than widely used. This provides ongoing disincentive for the industry to invest in their development, given the limited return on investment. Innovative ways of finding incentives to invest in further drug discovery are needed, and are currently being discussed on the global stage.

Valuing innovation: HTA practise and the impact for future medicines

Mr Mendel Grobler, Director, Patient Access, Pfizer Australia

This presentation will primarily deal with a largely unresearched aspect of Health Technology Assessment (HTA): its emerging impact on the future supply of new technology. Medical progress in the treatment of disease is dependent on innovation. In turn, innovation in any field is dependent on incentives. In the commercial world the incentive to commit capital to risky projects is the return on that capital, and in the modern world fiduciary responsibility requires the use of financial analytic techniques that explicitly balance risk with return. Now that HTA is beginning to influence the supply of future technology, it is both important and timely to consider how to use this influence to best serve society’s needs.